title: Outcome of pediatric acquired aplastic anemia: a developing world experience.
abstract: BACKGROUND
  Outcome data of children with acquired aplastic anemia (AA) are lacking from the developing world. Here, we describe the same from a centre in North India.
  
  
  METHODS
  Retrospective data regarding medical history, physical examination, complete blood count, bone marrow aspirate, and biopsy were retrieved for all children <18 years, with acquired AA admitted between January 2005 and June 2012. In addition, the outcome data after immunosuppressive therapy (IST) or bone marrow transplant (BMT) was obtained.
  
  
  RESULTS
  A total of 61 children were diagnosed with AA (Inherited-18 and acquired-43). Among 43 children with acquired AA, 3 had nonsevere and 40 had severe. One patient with nonsevere AA died of sepsis and 2 recovered spontaneously. Of the 40 remaining children with severe AA, 10 refused therapy and 3 died due to severe sepsis prior to any therapy. Five underwent upfront matched sibling donor BMT and one post-IST failure. Four year overall survival (OS) and event free survival (EFS) for children undergoing BMT was 100% and 80 ± 17.9, respectively. Out of 22 treated with IST, 20 were evaluable for response. Seventeen received one course and 3 received two course of IST. The overall response to IST was seen in 14/20 (70%). Only two achieved complete response while remaining 12 had partial response. The 4-year estimated OS and EFS for children treated with IST was 74.4 ± 12.1% and 65.6 ± 12.2.
  
  
  CONCLUSIONS
  Outcomes for children with AA are encouraging in the developing world although barriers like sepsis and treatment abandonment remain. BMT offers faster and complete recovery.
authors.affiliation: Pediatric Hematology Oncology & BMT Unit, Department of Pediatrics, Sir Ganga Ram Hospital , New Delhi , India.
authors.forename: Mohammed
authors.initials: M
authors.lastname: Ramzan
authors.forename: S P
authors.initials: SP
authors.lastname: Yadav
authors.forename: Mir Sadaqat Hassan
authors.initials: MS
authors.lastname: Zafar
authors.forename: Veronique
authors.initials: V
authors.lastname: Dinand
authors.forename: Anupam
authors.initials: A
authors.lastname: Sachdeva
mesh_terms: D000293:Adolescent; D064591:Allografts; D000741:Anemia, Aplastic / Q000401:mortality* / Q000601:surgery / Q000628:therapy; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003906:Developing Countries; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007194:India / Q000453:epidemiology; D008297:Male; D011239:Prednisolone / Q000627:therapeutic use; D012189:Retrospective Studies; D018805:Sepsis / Q000209:etiology / Q000401:mortality; D015996:Survival Rate; D013601:T-Lymphocytes / Q000276:immunology; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D011239:Prednisolone